Ocugen initiates Phase 3 clinical trial for OCU400 gene therapy for retinitis pigmentosa

Ocugen, Inc announced the first patient has been dosed in the Phase 3 LIMELIGHT clinical trial for OCU400, a novel gene therapy designed to treat retinitis pigmentosa (RP), a group of rare, genetic disorders that lead to progressive vision loss and blindness, according to a press release.

This marks the first time a gene therapy for RP with such a broad indication has reached this advanced stage of clinical testing.

The Phase 3 LIMELIGHT trial is informed by promising results from the Phase 1/2 study, which indicated positive trends in best-corrected visual acuity (BCVA), multi-luminance mobility testing (MLMT), and low-luminance visual acuity (LLVA) among treated eyes. Of note, 89% of RP participants in the Phase 1/2 trial demonstrated preservation or improvement in the treated eye on at least one of these measures. Specifically, 80% of subjects with RHO mutations showed either preservation or improvement in MLMT scores, and 78% of all subjects improved or maintained their MLMT scores.

The Phase 3 study will span 1 year and include 150 participants divided into 2 arms: 75 with RHO gene mutations and 75 gene-agnostic. Participants will be randomized in a 2:1 ratio to receive either the treatment (2.5 x 10^10 vg/eye of OCU400) or remain untreated. The trial is recruiting patients aged ≥8, ranging from early to late-stage RP.

A key focus of the study will be the luminance dependent navigation assessment, a more sensitive and specific functional measurement than MLMT used in prior trials. The primary endpoint is the proportion of responders in both treated and untreated groups achieving an improvement of at least 2 Lux levels from baseline.

The full press release can be found here.